CA3097375A1 - Polypeptides de capside d'aav modifies pour le traitement de maladies musculaires - Google Patents

Polypeptides de capside d'aav modifies pour le traitement de maladies musculaires Download PDF

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Publication number
CA3097375A1
CA3097375A1 CA3097375A CA3097375A CA3097375A1 CA 3097375 A1 CA3097375 A1 CA 3097375A1 CA 3097375 A CA3097375 A CA 3097375A CA 3097375 A CA3097375 A CA 3097375A CA 3097375 A1 CA3097375 A1 CA 3097375A1
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Prior art keywords
aav capsid
capsid polypeptide
aav
specified
muscle
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Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
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CA3097375A
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English (en)
Inventor
Dirk Grimm
Jonas WEINMANN
Josef EL ANDARI
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Universitaet Heidelberg
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Universitaet Heidelberg
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Publication of CA3097375A1 publication Critical patent/CA3097375A1/fr
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14123—Virus like particles [VLP]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La présente invention concerne un polypeptide de capside de virus adéno-associé (AAV) lié à un peptide de liaison comprenant une séquence d'acides aminés RGDX1X2 X3 X4, avec X1 à X4 étant des acides aminés sélectionnés indépendamment, pour une utilisation dans le traitement et/ou la prévention d'une maladie musculaire et/ou dans la régénération musculaire. La présente invention concerne en outre des polynucléotides, des cellules hôtes, des capsides de virus adéno-associés (AAV), des compositions pharmaceutiques, des utilisations et des procédés associés audit polypeptide de capside d'AAV.
CA3097375A 2018-04-27 2019-04-26 Polypeptides de capside d'aav modifies pour le traitement de maladies musculaires Pending CA3097375A1 (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18169822.6 2018-04-27
EP18169822 2018-04-27
PCT/EP2019/060790 WO2019207132A1 (fr) 2018-04-27 2019-04-26 Polypeptides de capside d'aav modifiés pour le traitement de maladies musculaires

Publications (1)

Publication Number Publication Date
CA3097375A1 true CA3097375A1 (fr) 2019-10-31

Family

ID=62167107

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3097375A Pending CA3097375A1 (fr) 2018-04-27 2019-04-26 Polypeptides de capside d'aav modifies pour le traitement de maladies musculaires

Country Status (7)

Country Link
US (1) US20210363193A1 (fr)
EP (1) EP3784288A1 (fr)
JP (2) JP2021521833A (fr)
CN (1) CN112040988A (fr)
AU (1) AU2019258830A1 (fr)
CA (1) CA3097375A1 (fr)
WO (1) WO2019207132A1 (fr)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022114243A1 (fr) * 2020-11-25 2022-06-02 Astellas Pharma Inc. Méthode de traitement de la dystrophie musculaire par ciblage du gène dmpk

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3856913A4 (fr) 2018-09-26 2022-10-26 California Institute Of Technology Compositions de virus adéno-associé pour une thérapie génique ciblée
MX2021005720A (es) 2018-11-16 2021-07-21 Astellas Pharma Inc Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina.
EP4031672A1 (fr) * 2019-09-20 2022-07-27 UCL Business Ltd Composition de thérapie génique et traitement de la cardiomyopathie arrythmogénique ventriculaire droite
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
CA3174863A1 (fr) 2020-04-14 2021-10-21 Ana BUJ BELLO Vecteurs pour le traitement d'une deficience en ceramidase acide
JP2023528580A (ja) * 2020-04-28 2023-07-05 ジェネトン 筋肉及び中枢神経系障害の遺伝子治療のための合成ａａｖカプシドの使用
WO2021230385A1 (fr) 2020-05-15 2021-11-18 Astellas Pharma Inc. Procédé de traitement de la dystrophie musculaire par ciblage d'un gène utrophine
MX2023001615A (es) 2020-08-07 2023-03-08 Spacecraft Seven Llc Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.
JP2023544683A (ja) 2020-09-10 2023-10-25 ジェネトン ペプチド改変されたａａｖカプシド
CN113754728B (zh) * 2020-09-30 2022-07-12 广州派真生物技术有限公司 腺相关病毒突变体及其应用
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
MX2023003984A (es) * 2020-10-09 2023-04-24 Tenaya Therapeutics Inc Metodos y composiciones para terapia genica con placofilina-2.
EP4244209A1 (fr) 2020-11-11 2023-09-20 European Molecular Biology Laboratory Particules virales modifiées destinées à la thérapie génique
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
WO2023060113A1 (fr) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions et procédés de production d'aav recombinants
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
AU2022420596A1 (en) * 2021-12-21 2024-07-11 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
US20230365968A1 (en) 2022-04-06 2023-11-16 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
US20230346977A1 (en) 2022-04-13 2023-11-02 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
EP4269426A1 (fr) * 2022-04-29 2023-11-01 Ospedale San Raffaele S.r.l. Thérapie génique
WO2023237748A1 (fr) 2022-06-10 2023-12-14 Genethon Capside d'aav modifiée par un peptide présentant une efficacité de transduction musculaire améliorée

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050106558A1 (en) * 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
US9677088B2 (en) * 2012-05-09 2017-06-13 Oregon Health & Science University Adeno associated virus plasmids and vectors
EP3384034B1 (fr) * 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Nouveaux capsides de virus adéno-associés recombinants avec du tropisme pour les muscles sqeletiques humains amélioré
TN2020000187A1 (en) * 2018-04-05 2022-04-04 Univ Devry Val Dessonne Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022114243A1 (fr) * 2020-11-25 2022-06-02 Astellas Pharma Inc. Méthode de traitement de la dystrophie musculaire par ciblage du gène dmpk

Also Published As

Publication number Publication date
EP3784288A1 (fr) 2021-03-03
CN112040988A (zh) 2020-12-04
JP2024020346A (ja) 2024-02-14
WO2019207132A1 (fr) 2019-10-31
AU2019258830A1 (en) 2020-12-03
US20210363193A1 (en) 2021-11-25
JP2021521833A (ja) 2021-08-30

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